Monthly News Roundup - February 2025

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on Feb 28, 2025.

FDA Updates Izervay Label to Lengthen Treatment Duration for Geographic Atrophy

Izervay (avacincaptad pegol) from Astellas is now FDA-approved without a limitation on duration of dosing, allowing greater flexibility when managing Geographic Atrophy (GA). Izervy is a complement inhibitor indicated for the treatment of GA secondary to age-related macular degeneration (AMD).

The recommended dose for Izervy is 2 mg (0.1 mL of 20 mg/mL solution) administered by intravitreal injection (injection into the eye) to each affected eye once monthly (about every 28 ± 7 days). Previously the regimen was only recommended to be given once monthly for up to 12 months.

Izervay treats geographic atrophy (GA), the advanced stage of age-related macular degeneration (AMD) and helps slow down vision deterioration, allowing patients to maintain better vision for a longer period of time. Geographic atrophy can lead to irreversible vision loss.

Geographic atrophy begins as small damaged areas in the retina, gradually expanding and affecting central vision, which is essential for sharp, detailed sight. This damage is primarily caused by an overactive immune response known as the complement system.

Izervay contains the active ingredient avacincaptad pegol which works by targeting C5 to decrease activity of the complement system and potentially slow the progression of geographic atrophy.

Approval was based on positive results from the GATHER2 Phase 3 clinical trial, which evaluated the efficacy and safety of Izervay through year two. Results showed that Izervay continued to reduce the rate of GA lesion growth in patients with GA secondary to AMD through two years versus sham, with benefit appearing as early as 6 months and more than doubled over two years compared to year one.

The most common adverse reactions were bleeding in the conjunctiva of the eye (13%), increased pressure inside the eye (9%), blurred vision (8%) and neovascular age-related macular degeneration (macular degeneration where abnormal blood vessels grow underneath the retina, 7%). Eye injections like Izervay can cause an eye infection (endophthalmitis) or separation of layers of the retina (retinal detachment).

Izervay, from Astellas Pharma Inc, was first approved for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) on August 4, 2023.

FDA Approves Genentech’s Evrysdi Tablet for Spinal Muscular Atrophy (SMA)

Genentech has announced the FDA approval of Evrysdi (risdiplam) tablet for adults and children living with spinal muscular atrophy (SMA). Evrysdi 5 milligram (mg) tablet can either be swallowed whole or dispersed in water. Evrysdi was first available as a liquid solution given via a provided oral syringe. The Evrysdi oral solution will remain available for those on other doses of Evrysdi and for those who may prefer the oral solution.

Evrysdi is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival of motor neuron (SMN) protein deficiency. It is the only non-invasive disease-modifying treatment for SMA.

The new tablet is suitable for people 2 years of age or older who weigh more than 44 lbs (20 kgs). The dose is one 5 mg tablet orally once daily. Swallow the tablets whole with water; do not chew, cut, or crush.

The Evrysdi tablet is the first tablet available for SMA. Approval was based on the results of a bioequivalence study, which demonstrated that the 5 mg tablet, whether swallowed whole or dispersed in non-chlorinated, filtered drinking water provides comparable exposure to the original risdiplam oral solution.

Evrysdi liquid, from Genentech, was first approved on August 7, 2020 for treatment of Spinal Muscular Atrophy (SMA) in people 2 months and older.

FDA Approves Gomekli, a MEK Inhibitor to Treat Genetic Neurofibromatosis (NF1-PN)

The FDA has cleared Gomekli (mirdametinib), a MEK inhibitor for the treatment of adults and children 2 years of age and older with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) who have symptomatic plexiform neurofibromas not amenable to complete surgical removal.

Neurofibromatosis type 1 (NF1) is a rare genetic disorder with mutations in the NF1 gene. NF1 patients are at risk of developing plexiform neurofibromas, which are tumors that grow along the peripheral nerve sheath and can cause severe disfigurement, pain and functional impairment. Plexiform neurofibromas can become malignant and potentially fatal.

Gomekli contains the active ingredient mirdametinib which inhibits protein kinase 1 and 2 (MEK1/2) and downstream phosphorylation of ERK, which has been shown to reduce neurofibroma tumor volume and proliferation in a mouse model of NF1.

Approval was based on results from the Phase 2b ReNeu trial of 58 adults and 56 pediatric patients with NF1-PN, which showed that Gomekli treatment resulted in an objective response rate (ORR), the primary endpoint, of 41% (N= 24/58) in adults and 52% in children (N=29/56). The median best reductions in tumor volume were -41% (range: -90 to 13%) in adults and -42% (range: -91 to 48%) in children.

Gomekli capsules and tablets for oral suspension are given by mouth twice daily, with or without food, for the first 21 days of each 28-day cycle until the disease worsens or unacceptable side effects occur.

Warnings and precautions associated with Gomekli include ocular (eye) toxicity, left ventricular dysfunction (trouble with heart’s ability to pump blood), skin side effects including rash, and embryo-fetal toxicity.

Common adverse reactions (at least 25%) in adults included rash, diarrhea, nausea, muscle / bone pain, vomiting, and fatigue. In children, adverse reactions included rash, diarrhea, musculoskeletal pain, stomach pain, vomiting, headache, paronychia (infection of skin around the nail), left ventricular dysfunction (trouble with heart’s ability to pump blood), and nausea.

Gomekli is manufactured by SpringWorks Therapeutics, Inc. and received a rare pediatric disease priority review voucher from the FDA.

FDA Approves Onapgo to Treat Motor Fluctuations in Adults with Advanced Parkinson’s Disease

In February the FDA cleared Onapgo (apomorphine hydrochloride) as the first subcutaneous apomorphine infusion device for the treatment of motor fluctuations (OFF episodes) in adults with advanced Parkinson’s disease (PD). As the motor symptoms of PD worsen, patients often experience states between ON (when their medication is working), and OFF (when it’s not working optimally). Onapgo is manufactured by Supernus Pharmaceuticals.

Apomorphine is believed to work as a treatment for PD by stimulation of post-synaptic dopamine D2-type receptors within the brain. Apomorphine works as a dopamine agonist that binds with high affinity to the dopamine D4 receptor, and moderate affinity for the dopamine D2, D3, and D5, and adrenergic α1D, α2B, α2C receptors.

Apomorphine was first approved in 2004 under the brand name Apokyn to treat acute, intermittent OFF episodes in Parkinson’s disease. Onapgo now provides a continuous subcutaneous infusion of apomorphine through an infusion device utilizing a small and lightweight wearable device.

The approval is based on results from the TOLEDO study, a Phase 3, 12-week, double-blind, randomized, placebo-controlled study (N=107). The primary efficacy endpoint, based on patient diaries, was the mean change in total daily OFF time. Onapgo significantly reduced the amount of daily OFF time at 12 weeks from baseline (p=0.0114), with Onapgo-treated patients (n=53) experiencing a 2.6-hour reduction compared to placebo (n=51) of 0.9 hours. In addition, key secondary endpoints vs. placebo, such as daily GOOD ON time (p=0.0188) and Patient Global Impression of Change (PGIC), p<0.0001 were met. Results were seen as early as week one.

Apokyn is given by subcutaneous injection with a multiple-dose pen injector that can be used up to 5 times a day, with doses no less than two hours apart. Doses are generally administered over the waking day (e.g., 16 hours).

The most common adverse reactions (in at least 10% of patients) were infusion site nodule, nausea, drowsiness, infusion site redness/swelling, dyskinesia (abnormal movements), headache, and trouble sleeping.

Onapgo is expected to be available in the second quarter of 2025.

FDA Approves AbbVie’s Emblaveo for Complicated Intra-Abdominal Infections

AbbVie has announced the approval of Emblaveo (aztreonam and avibactam), a monobactam antibacterial and beta-lactamase inhibitor intravenous (IV) combination approved for use in people 18 years and older for the treatment of complicated intra-abdominal infections. Emblaveo contains a 3:1 fixed ratio combination of the monobactam antibacterial aztreonam and the beta-lactamase inhibitor avibactam.

Complicated intra-abdominal infections extend beyond the hollow viscus of origin into the peritoneal space and are associated with either abscess formation or peritonitis.

Emblaveo is approved in combination with metronidazole, for patients 18 years and older who have limited or no alternative options for the treatment of complicated intra-abdominal infections (cIAI), including those caused by the following susceptible Gram-negative microorganisms: Escherichia coli, Klebsiella pneumoniae, Klebsiella oxytoca, Enterobacter cloacae complex, Citrobacter freundii complex, and Serratia marcescens.

FDA approval of Emblaveo was supported by prior findings regarding the efficacy and safety of aztreonam for the treatment of cIAI, and clinical trial results from the Phase 3 REVISIT study.

Emblaveo is administered by intravenous infusion over 3 hours. A loading dose is followed by maintenance doses every 6 to 12 hours (based on creatinine clearance) for 5 to 14 days.

Common adverse reactions (in at least 5% of patients) include liver adverse reactions, anemia, diarrhea, hypokalemia (low potassium blood levels) and pyrexia (fever).

Emblaveo is manufactured by AbbVie.

FDA Approves the Miudella Copper IUD to Prevent Pregnancy

In February, the FDA approved Miudella, a copper-containing intrauterine device (IUD) indicated for up to 3 years use for prevention of pregnancy in females of reproductive potential. Miudella is manufactured by Sebela Pharmaceuticals, Inc.

Miudella is a hormone-free, low-dose copper IUD contraceptive device with a flexible frame made of nitinol, a material that has superelastic properties. This allows placement of the copper to achieve similar efficacy to the currently available copper IUD (brand name: Paragard) with less than half the dose of copper.

Miudella was studied in 3 clinical trials in women aged 17 to 45 years. The Phase 3 study had a primary endpoint of contraceptive efficacy through 3 years of use as assessed by the Pearl Index (defined as the number of pregnancies per 100 women over one year). In the efficacy cohort from the Phase 3 study (n=1397), the first-year Pearl Index was 0.94 (95% CI, 0.43-1.78) and the cumulative 3-year Pearl Index was 1.05 (95% CI, 0.66-1.60) or 99% efficacy. Both clinicians and study participants also reported positive experiences with placement of Miudella, with an overall placement success rate of 98.8%.

Miudella is inserted into the fundus of the uterine cavity, and must be removed or replaced after 3 years.

Miudella comes with a Boxed Warning for complications due to improper insertion. Miudella is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Miudella REMS program to ensure healthcare providers are trained on the proper insertion of Miudella prior to first use.

Warnings and precautions associated with Miudella include risk of ectopic pregnancy; increased risk of spontaneous abortion, septic abortion, premature delivery, sepsis, septic shock, and death if pregnancy occurs; pelvic inflammatory disease; perforation resulting in embedment or translocation; partial or complete expulsion; and altered bleeding patterns.

Common adverse reactions (incidence ≥ 5%) include heavy menstrual bleeding, dysmenorrhea, intermenstrual bleeding, pelvic discomfort, procedural pain, pelvic pain, post procedural hemorrhage, dyspareunia.

Miudella will be available to patients through trained healthcare providers in 2025.

Two New Denosumab Biosimilars Approved: Ospomyv and Xbryk

In February, the FDA approved Ospomyv (denosumab-dssb, 60 mg pre-filled syringe), a biosimilar referencing Prolia, and approved Xbryk (denosumab-dssb; 120 mg vial), a biosimilar referencing Xgeva. A biosimilar is a biologic medication that is highly similar to a biologic already approved by FDA (also called the reference product). Biosimilars have no clinically meaningful differences from the reference product with the same safety and effectiveness.

Ospomyv is approved for the treatment of postmenopausal women with osteoporosis at high risk for fracture, treatment to increase bone mass in men with osteoporosis at high risk for fracture, and treatment of glucocorticoid-induced osteoporosis in men and women at high risk for fracture, among other uses.

Xbryk is approved for the prevention of skeletal-related events in patients with multiple myeloma and in patients with bone metastases from solid tumors, treatment of adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity, and for the treatment of hypercalcemia of malignancy refractory to bisphosphonate therapy.

Both Ospomyv and Xbryk are RANK ligand (RANKL) inhibitors that work by blocking the formation and activity of osteoclasts, the cells responsible for breaking down bone tissue. The FDA approval was based on totality of evidence including analytical, non-clinical data, and clinical data.

Common side effects of denosumab may include bladder infection (painful or difficult urination); lung infection (cough, shortness of breath); headache; back pain, muscle or joint pain; increased blood pressure; cold symptoms such as stuffy nose, sneezing, sore throat; high cholesterol; or pain in your arms or legs.

Ospomyv (denosumab-dssb) is the second FDA-approved biosimilar to Prolia (denosumab) after the approval of Jubbonti (denosumab-bbdz) in 2024.

Xbryk (denosumab-dssb) was also approved as the second biosimilar to Xgeva (denosumab) after the approval of Wyost (denosumab-bbdz) in 2024.

Ospomyv and Xbryk are manufactured by Samsung Bioepis.

FDA Approves Merilog (insulin-aspart-szjj), a Biosimilar to NovoLog

The FDA has approved Merilog (insulin aspart-szjj), a rapid-acting human insulin analog biosimilar to NovoLog (insulin aspart) indicated to improve glycemic (blood sugar) control in adults and children with diabetes mellitus. Merilog is the first FDA-approved biosimilar to NovoLog.

Merilog is biosimilar to NovoLog but does not have an interchangeability designation at this time, meaning the product may be substituted for the reference product (in this case, Novolog) without approval by the prescriber.

FDA approval of Sanofi’s Merilog was based on data that demonstrated it is biosimilar to NovoLog. It is administered as a subcutaneous (under the skin) injection and is supplied as 100 units/mL (U-100) of insulin aspart-szjj in a 10 mL multiple-dose vial and 3 mL single-patient-use prefilled pen.

Warnings and precautions include hyperglycemia or hypoglycemia (high or low blood sugars) with changes in insulin regimen, low blood sugar which may be life-threatening, hypersensitivity (allergic) reactions, and hypokalemia (low potassium levels).

Adverse reactions observed with insulin aspart products include hypoglycemia, allergic reactions, local injection site reactions, lipodystrophy (redistribution of body fat), rash, and pruritus (itching).

FDA Approves Penmenvy, a Pentavalent Meningococcal Vaccine

Penmenvy (meningococcal groups A, B, C, W, and Y vaccine) from GSK is a newly approved vaccine indicated for active immunization to prevent Invasive Meningococcal Disease (IMD) caused by the bacteria Neisseria meningitidis serogroups A, B, C, W, and Y in individuals 10 through 25 years of age.

Invasive Meningococcal Disease (IMD) is an uncommon but serious illness that, despite treatment, can lead to death in as little as 24 hours. About 1 in 5 survivors may experience long-term consequences such as brain damage, amputations, hearing loss, and nervous system problems. People between the ages of 16 and 23 years are at high risk due to common behaviors that help transmit the bacteria such as living in close quarters (for example: college dormitories).

Penmenvy combines the antigenic components of Bexsero (MenB vaccine) and Menveo (MenACWY vaccine). Penmenvy is a pentavalent MenABCWY vaccine that provides broad serogroup coverage in one vaccine, reducing the total number of injections required to protect against invasive meningococcal disease. It protects against invasive meningococcal disease by complement-mediated antibody-dependent killing of Neisseria meningitidis serogroups A, B, C, W, and Y.

FDA approval was supported by positive results from 2 phase III trials which evaluated the vaccine’s safety, tolerability, and immune response in over 4,800 participants aged 10-25 years.

Bexsero was first approved in 2015 for the prevention of IMD caused by Neisseria meningitidis serogroup B in individuals aged 10 through 25 years.

Menveo was first approved in 2010 for the prevention of IMD caused by Neisseria meningitidis serogroups A, C, Y, and W in individuals aged from 2 months through 55 years of age.

Penmenvy is administered by intramuscular (IM) injection as 2 doses, spaced 6 months apart.

Warnings and precautions include syncope (fainting). Administration should take place where an injury from fainting can be avoided.

Commonly reported adverse reactions (in at least 10% of the population) after Dose 1 and Dose 2, respectively in individuals aged 10 through 25 years include pain at the injection site (92% and 88%), fatigue (51% and 42%), headache (42% and 36%), myalgia / muscle pain (15% and 12%), nausea (15% and 10%), erythema / redness (13% and 12%), and swelling (13% and 12%).

FDA Approves Romvimza for the Treatment of Symptomatic Tenosynovial Giant Cell Tumor

The FDA has cleared Romvimza (vimseltinib) for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) in which surgery will potentially cause worsening functional limitation or a more severe health outcome.

Tenosynovial giant cell tumor (TGCT) is a rare, typically non-cancerous tumor that develops inside or near the joints, often in fingers, knees, elbows or wrists. It is thought to be caused by translocation of chromosomes, when chromosomes (part of your DNA) break apart and reconnect in new pairs. Although benign, the tumors can grow and cause damage to the body causing pain, swelling, and limitation of joint movement.

Romvimza is a switch-control kinase inhibitor of colony stimulating factor 1 receptor (CSF1R) and works in the treatment of TGCT by selectively and potently inhibiting CSF1R.

FDA approval was supported by data from the pivotal Phase 3 MOTION study, where Romvimza met the primary endpoint of improved objective response rate (ORR) compared to placebo. All key secondary endpoints were also met with statistically significant and clinically meaningful improvements in quality-of-life measures and a well-tolerated safety profile.

Romvimza capsules are administered orally twice weekly, with a minimum of 72 hours between doses. Take Romvimza on the same days of each week. It comes as 14 mg, 20 mg, and 30 mg oral capsules.

Warnings and precautions associated with Romvimza include liver toxicity, embryo-fetal toxicity (harm to an unborn baby), allergic reactions to FD&C Yellow No. 5 (tartrazine) and No. 6 (Sunset Yellow FCF), and increased serum creatinine (without affecting kidney function).

Common adverse reactions (in at least 20% of patients in studies) include periorbital edema (swelling of tissues around the eye), fatigue, rash, fluid retention in face or extremities, pruritus (itching) and laboratory abnormalities (increased liver enzymes, increased cholesterol, decreased white blood cells).

Romvimza is from Deciphera Pharmaceuticals.

FDA Approves Ctexli for Cerebrotendinous Xanthomatosis, a Rare Bile Acid Disorder

In February, the FDA approved Ctexli (chenodiol) tablets, a synthetic form of the bile acid chenodeoxycholic acid indicated for the treatment of cerebrotendinous xanthomatosis in adults. Ctexli is the first treatment containing chenodiol that is specifically indicated to treat CTX.

Cerebrotendinous xanthomatosis (CTX) is a rare, genetic bile acid synthesis disorder caused by a mutation in the CYP27A1 gene. CTX is characterized by the accumulation of deposits of cholesterol and cholestanol the nerve cells and membranes potentially causing damage to the brain, spinal cord, tendons, lens of the eye and arteries.

Ctexli works by replacing deficient levels of chenodeoxycholic acid to reduce abnormal deposits of the cholesterol metabolites thought to be responsible for clinical abnormalities in CTX.

Chenodiol, the active ingredient in Ctexli, was first approved under the brand name Chenix in 1983 and reapproved as Chenodal in 2009 for the treatment of gallstones

FDA approval for Ctexli was supported by data from a clinical study which showed that patients treated with Ctexli demonstrated a significant reduction in plasma cholesterol metabolites (cholestanol and urine 23S-pentol) compared to patients treated with placebo (a pill that contains no medicine).

Ctexli tablets are administered by mouth three times daily.

Warnings and precautions associated with Ctexli include hepatotoxicity. Baseline liver transaminase (alanine aminotransferase [ALT] and aspartate aminotransferase [AST]) and total bilirubin levels should be obtained prior to initiating treatment with Ctexli.

Common adverse reactions (in at least 14% of people in studies) include diarrhea, headache, abdominal (stomach) pain, constipation, hypertension (high blood pressure), muscular weakness, and upper respiratory tract infection.

Ctexli is manufactured by Mirum Pharmaceuticals Inc.

Posted : 2025-03-03 06:00

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Monthly News Roundup - February 2025

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